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July 2, 2018 at 9:33 am in reply to: All-Time Career Sack Leaders, Updated with John Turney's pre-1982 Numbers #87779
nittany ram
Moderator6 Rams on the list, including 3 in the top 5.
Jones, 3rd (173.5)
Greene, 4th (160)
JY, 5th (150.5)
Bacon, 21st (130)
Carter, 43rd (104.5)
Dryer, 46th (103)Leslie O’Neal is on the list tied at #19. He was a Ram briefly.
nittany ram
ModeratorMy deviled eggs have been described as “transcendent”.
Mostly by me.
nittany ram
ModeratorThe Rams are going to be a nice team this year. They will be in the playoffs. Book it.
Just making the playoffs isn’t good enough this year.
Anything less than a deep playoff run (NFC Championship game) would be a disappointment.
Maybe not “Columbia losing in the World Cup in 94” level disappointment (the player that cost them the match was murdered).
But then again, maybe so.
nittany ram
ModeratorThe only reason you would have to fear gluten is if you have celiac disease.
Gluten is most likely harmless for everyone else.
Link: https://theness.com/neurologicablog/index.php/non-celiac-gluten-sensitivity/
About a third of Americans report that they are trying to reduce or avoid gluten in their diet. If Jimmy Kimmel’s funny stunt is any indication, most probably don’t know what gluten even is. The gluten-free diet has officially become a fad, and “gluten” has been tagged as something vaguely bad that should be avoided.
About 1% of people have a disease called Celiac, which is an autoimmune reaction to gluten. This is a serious disease that can make people very ill if they consume even the smallest amount of gluten. A diagnosis of Celiac can be confirmed with an antibody test (anti-gliadin antibodies), or, if necessary, a stomach biopsy.
Gluten is a composite protein composed of two parts, gliadin and glutenin. It is found in wheat, rye, barley, spelt, and related grains. It is a springy protein that gives bread its elasticity. Celiac disease is an immune reaction to the gliadin part of the protein.
Celiac is fairly well understood and is non-controversial. What is controversial is a disorder known as non-celiac gluten sensitivity (NCGS) – believed to be an intolerance to gluten that causes gastrointestinal symptoms. NCGS is controversial, and in fact may not truly exist at all.
NCGS should be considered a hypothesis, not a confirmed entity, but this has not stopped self-diagnosis and treatment from becoming popular.
It is sometimes tricky to confirm whether or not a new possible diagnosis truly exists, or if it is just a misdiagnosis of other diseases and disorders. Diseases are usually first recognized by their clinical syndrome, and then later investigation uncovers the cause or pathophysiology of the disease. Often at this stage, when we discover what is happening biologically, diseases are reclassified, and diagnoses are sometimes combined, and other times split apart.
There are some diagnoses, however, that live on the fringe, never gaining scientific support. Throughout history, it seems, there have always been faddish diagnoses used as popular labels for common symptoms. At the turn of the 19th century “neurasthenia” was a common label for vague or common symptoms. In the mid 20th century syphilis (although a real disease) was often used as a convenient diagnosis for any unexplained symptoms.
More recently we have chronic Lyme, candida hypersensitivity, multiple chemical sensitivity, electromagnetic sensitivity, and a host of other vague syndromes.
Electromagnetic sensitivity is similar to NCGS in that both are believed to be a sensitivity with symptoms resulting from a specific exposure. In both cases, therefore, we can address the core question (does the sensitivity exist) by studying blinded exposures. In the case of electromagnetic sensitivity, when properly blinded those who believe they have this condition cannot detect exposure.
What about NCGS? It has not been established that NCGS exists, or that people who believe they have this condition actually are responding physiologically to gluten. There are two possibilities that need to be carefully considered. The first is that perceived gluten sensitivity is an observational artifact, a type of nocebo effect. GI symptoms are notoriously sensitive to mood and expectation. There are also generic biases such as confirmation bias that can lead to the perception of false associations.
It is still not clear, in other words, that there is an actual association between consuming gluten and GI symptoms. Individuals may firmly believe that they have such an association, but we know from countless historical examples and experiments that such firm beliefs can form in the absence of a true association.
The second possibility that needs to be seriously considered is that in some people who are self-diagnosed with NCGS, they are reacting to something else that is common in gluten-containing foods. If this is the case, then gluten is an innocent bystander. This would be very important to discover, for obvious practical reasons.
A recent study suggests that this might be the case. Biesiekierski et. al. did a well controlled series of studies in which they challenged subjects with possible NCGS with carefully controlled diets with various amounts of gluten. They found no association between gluten consumption and reported symptoms, arguing very strongly against NCGS as a real entity.
Their study did, however, suggest another possible culprit – FODMAPs (fermentable, oligo-, di-, monosaccharides, and polyols). These are also common in breads and other foods containing gluten. In the study subjects, GI symptoms improved when FODMAPs and gluten were removed, but then reintroducing gluten had no association with return of symptoms. The authors conclude:
In a placebo-controlled, cross-over rechallenge study, we found no evidence of specific or dose-dependent effects of gluten in patients with NCGS placed diets low in FODMAPs.
They were not, however, testing whether or not FODMAPs were a cause of GI symptoms, and so cannot conclude if this is the true cause. A follow up study would need to be done to verify that (perhaps we’ll see a FODMAP-free fad before this science can be done). If true it would explain why some people do have reduction in GI symptoms when they avoid gluten, because they are also avoiding FODMAPs.
Conclusion
The best evidence we currently have suggests that NCGS is probably not a real entity. Blinded challenges do not show any correlation, and there is currently no evidence for a specific mechanism. Those who are self-diagnosed with NCGS probably fall into one of three categories:
1- Borderline true Celiac disease (a small minority that can be sorted out with diagnostic tests)
2- GI symptoms due to non-dietary reasons with a false association with gluten due to confirmation bias and nocebo effects
3 – GI symptoms due to some other food exposure. FODMAPs are one possibility, but more research needs to be done.
The real risk of the gluten-free fad is that it distracts from what is really going on. Popular diagnoses (whether real or not) do tend to attract self-diagnosis, and become an impediment to a more proper diagnosis. There is a tendency to prematurely settle on the popular diagnosis, and then fail to consider all possibilities.
In the case of NCGS, there may be something else in food to which some people are sensitive. Or, diet may not be the answer at all.
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This reply was modified 6 years, 10 months ago by
nittany ram.
nittany ram
ModeratorCool shot of my dad John Hock #63. pic.twitter.com/2OoZTD0pYm
— Jim Hock (@jimhock) June 24, 2018
nittany ram
Moderator“He’s the head of a country, and I mean he’s the strong head,” Trump said. “He speaks and his people sit up at attention. I want my people to do the same.”
Yeah, he said it. He doesn’t even try to hide his desire to be the Supreme Ruler of America.
His minions are all for it. 52% of Republicans polled said they would gladly support postponing the 2020 election if Glorious Leader asked them to.
nittany ram
ModeratorThat’s shocking.
==========
Well zooey, dont even get me started on salamanders.
The MSM is totally silent on the Salamander ban.
==============Killer Fungus news:https://earther.com/scientists-call-for-total-ban-on-amphibian-imports-to-s-1820976014
America has the highest salamander biodiversity of any country in the world. But that incredible natural heritage is threatened by a killer fungus that has already decimated salamander populations in Europe. Now, a dozen scientists who study amphibians and conservation are calling for a total ban on amphibian imports into the United States in an effort to prevent a mass die-off of our nation’s salamanders…
w
vDon’t worry. Trump’s tariff on European imports will slow the amphibian trade to a trickle.
Of course, his roll back on environmental regulations (like allowing coal companies to dump waste water into waterways) probably won’t help quite as much…
nittany ram
ModeratorI have to say how much I appreciate and admire zooey’s posts on Facebook concerning these issues.
To use the parlance of today’s generation, he is “totes killing it”.
nittany ram
ModeratorTodd Gurley is 23, yet he was excluded. That calls the validity of the entire list into question. There needs to be an investigation with charges pending.
According to this video, the Saints have 4 or 5 of the top players under 25.
nittany ram
ModeratorWell but to me thats like saying Orcs should be more like elves. Ya know.
w
vWell, if you consulted your Tolkien, you’d see that orcs were decended from elves; their bodies and minds twisted by the evil Melkor…
The point being, the orcs were forced to be who and what they are. They had no choice.
Unlike the orcs, the dems have a choice – they just choose to be greedy.
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All of that reminds me of this quote:
“There are two novels that can change a bookish fourteen-year old’s life: The Lord of the Rings and Atlas Shrugged. One is a childish fantasy that often engenders a lifelong obsession with its unbelievable heroes, leading to an emotionally stunted, socially crippled adulthood, unable to deal with the real world. The other, of course, involves orcs.”
[Kung Fu Monkey — Ephemera, blog post, March 19, 2009]”
― John Rogers
That’s a good quote, Billy.
nittany ram
ModeratorWell but to me thats like saying Orcs should be more like elves. Ya know.
w
vWell, if you consulted your Tolkien, you’d see that orcs were decended from elves; their bodies and minds twisted by the evil Melkor…
The point being, the orcs were forced to be who and what they are. They had no choice.
Unlike the orcs, the dems have a choice – they just choose to be greedy.
nittany ram
ModeratorThe Top 100 Players of 2018 revealed players ranked No. 20-11 last night. Here they are pic.twitter.com/YMywOkyxRW
— NFL Media (@NFLMedia) June 19, 2018
That doesn’t look right.
I’m pretty sure the Rams have more than 10 players.
nittany ram
ModeratorGawd-damn. I defy anyone to watch that and not bawl.
nittany ram
Moderator“As far as this summit goes, Trump DID get something.”
You Trump supporters are all the same. You make bold proclamations about all that he is accomplishing, but when pressed on specifics you can only reply with “something” or “this n’ that”.
BTW, now that you’ve been outed as a closet Trump supporter, your new board handle will be “MAGA Scaramucci Ram”.
nittany ram
Moderator“A monopoly is forming in agrochemicals and GMO crop species that will leave the farmers of the world with no real choices about how to grow their crops if they want to be profitable…..”
Well, I’m not going to defend the business practices of Monsanto or Bayer, but if farmers around the world were forced to use GM crops we would see better outcomes in terms of costs, sustainability, yield, land use efficiency, nutrition, environmental impact…
——————
Well i hear Monsanto genetically modified tower number 7. And thats why it wilted like Bulger in 2009.
w
vStrange system we’ve concocted for ourselves.
The motivations of corporations can’t be trusted because their wants might not align with my needs, yet I am fully dependent on them for my needs.
For example, pharmaceutical companies can’t be trusted, but my wife is only alive because of pharmaceutical companies. So we are beholden to them.
So we create government agencies to watch over corporations, but then we allow the corporations to influence the people in the government who run those agencies.
The extent that logic must be contorted in support of capitalism is truly amazing.
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This reply was modified 6 years, 10 months ago by
nittany ram.
nittany ram
ModeratorBernie Sanders Is Losing Primary Battles, But Winning A War
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June 8, 20185:00 AM ET
Headshot of Scott Detrow, 2018
SCOTT DETROWTwitter
Sen. Bernie Sanders, I-Vt., greets the crowd as he arrives for a campaign rally in Tulsa, Okla., in 2016, when he built a large political movement during his bid for the Democratic presidential nomination. Many in the party have adopted parts of his agenda, even though candidates he backs mostly have lost in 2018.
Jacquelyn Martin/AP
Since most of the congressional candidates that Vermont Sen. Bernie Sanders endorsed this year are losing contested primaries, then Sanders’ political clout must be fading, right?“That’s a stupid argument,” Sanders told NPR this week.
“You know, he has a much broader look at politics than just elections,” Sanders’ longtime strategist Jeff Weaver said.
That is evident. The 2016 candidate repeatedly questioned the political value of his endorsements, and even expressed some mild indifference to the race-by-race results of the primaries he’s waded into.
Democrats Breathe Sigh Of Relief After Tuesday’s Primaries
ANALYSIS
Democrats Breathe Sigh Of Relief After Tuesday’s Primaries
Sanders’ broader goal is to get more first-time voters and first-time candidates involved in the political process, and to keep pushing progressive policies like a Medicare-for-all health care plan into the Democratic mainstream.If that takes more than one election cycle, so be it.
“I hope they win,” Sanders said. “Maybe they don’t. But if you get 45 percent of the vote now, next time you may well win.”
In the U.S. House primaries that have happened so far, Sanders has endorsed six candidates in contested races. Only two of his chosen House candidates have won contested primaries, and one was an incumbent: Rep. Nanette Barragán of California.
Even if many of his hand-picked candidates are coming up short, more of the Democrats who are winning are lining up closer to Sanders anyway. A Medicare-for-all, single-payer health care plan continues to gain support among Democratic candidates, and the $15 minimum wage Sanders made a key part of his presidential campaign has been adopted as a cause by party leaders across the country.
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Likely 2020 Democratic Candidates Want To Guarantee A Job To Every American
POLITICS
Likely 2020 Democratic Candidates Want To Guarantee A Job To Every American
But given his broad success at reshaping the party, the question lingers as to why so many of the candidates bearing Sanders’ personal seal of approval are losing.This week, another Sanders-endorsed House candidate lost a Democratic primary by a wide margin. Pete D’Alessandro ran Sanders’ Iowa campaign in 2016, which resulted in a virtual tie with Hillary Clinton. This year, the Vermont senator campaigned alongside him, cut a TV commercial for him and helped him raise money. But D’Alessandro finished in third.
“I could be 100 percent in terms of my endorsements,” Sanders told NPR. “All you’ve got to do is endorse establishment candidates who have a whole lot of money, who are 40 points ahead in the poll. You know what, you’ll come and say, ‘Bernie, you were 100 percent supportive of these candidates, they all won.'”
“The candidates that we support, by and large with few exceptions, are all candidates who are taking on the establishment, and are often outspent,” he added.
Sanders and Weaver argue that a race-by-race accounting isn’t the best way to track what the 2016 Democratic presidential runner-up is doing this year.
“The issue here is not that I think a Bernie Sanders, or frankly the endorsement of anybody else, is some magical potion to get people elected,” Sanders said. “Frankly, between you and me, I’m not sure how much endorsements – how significant they are. Sometimes they help, sometimes not much.”
Two of the 17 candidates Sanders has backed this year say that, in their minds, there’s no question the endorsement helped.
“It did a lot of good for our campaign,” said Greg Edwards, who ran in a crowded Pennsylvania House primary last month. “It increased my name ID, helped me get volunteers, helped with fundraising, certainly. And we got a lot of media attention out of it. I think we got four or five press hits.”
Still, Edwards also ended up third in his race.
But Edwards centered his campaign around policies many voters now associate with Sanders. “Universal health care, Medicare-for-all, around universal preschool, around debt-free college. Around increasing the minimum wage from $7.25 to $15,” he said.
“Many of these issues were considered fringe issues, and now they are mainstream issues that we take for granted that there, of course, are legions of Democratic candidates running on those platforms,” said Jeff Weaver. “Three or four years ago you would not have seen candidates running on that platform I would have considered to be outside the mainstream.”
Bernie Sanders Knows His Medicare-For-All Bill Won’t Pass. That’s Not The Point
POLITICS
Bernie Sanders Knows His Medicare-For-All Bill Won’t Pass. That’s Not The Point
When Sanders introduced his latest single-payer health care bill last year, it was quickly endorsed by several of the Senate Democrats mentioned as possible 2020 presidential candidates.Braddock, Pa., Mayor John Fetterman was running on many of those issues two years ago, when he entered Pennsylvania’s Democratic U.S. Senate primary against two better-funded candidates.
Fetterman had hoped for a Sanders endorsement that year, but didn’t get it. Fetterman finished third in that 2016 race.
The call finally came two years later, when Fetterman was running for lieutenant governor in a crowded field that included an incumbent. “I don’t care who you are, but you know when somebody says, ‘Please hold for Senator Sanders,’ and then you hear his voice on the line say, ‘Hi John’, you’re like, ‘Oh my gosh!'”
“What I ran on in 2016, that didn’t change in 2018,” said Fetterman, who won last month’s primary. But now, many other Democratic candidates share those views, too.
“A Democrat can’t be elected nationally if he or she doesn’t embrace his idea of Medicare-for-all, I fundamentally believe that,” said Fetterman. “And at the heart of that, that’s what Sen. Sanders has always championed.”
nittany ram
Moderator“A monopoly is forming in agrochemicals and GMO crop species that will leave the farmers of the world with no real choices about how to grow their crops if they want to be profitable…..”
Well, I’m not going to defend the business practices of Monsanto or Bayer, but if farmers around the world were forced to use GM crops we would see better outcomes in terms of costs, sustainability, yield, land use efficiency, nutrition, environmental impact…
nittany ram
Moderator<
That’s the stuff I don’t buy. The science on this is pretty clear I think.…
================
Well i used to think it was clear, but now i have doubts. There are plenty of sciency people that dont buy the official version. And i do not have the background to know. So…I have to ‘trust’…who? I dunno.
So thats where I’m at.
And Mack has gone awol.
w
vWhat we do have is a world full of thousands of independent engineers, metallurgists, chemists, chemical engineers, and so on (how many other fields?) not just in the USA but all over the world, and they all have all the data they need to reach conclusions.
Is there a universal outcry from discipline-relevant scientists in american universities and around the world that something is fishy about the accepted story of what happened to the buildings? No.
Instead what you constantly find is things like this:
Did a European Scientific Journal Conclude 9/11 Was a Controlled Demolition?
https://www.snopes.com/fact-check/journal-endorses-911-conspiracy-theory/
Viral news stories misattributed an article written by “9/11 Truth” advocates as one published in the European Scientific Journal; it was actually published in Europhysics News, which is not peer-reviewed.
A conspiracy of this magnitude would have to involve 1000s of people. It would nearly be impossible to keep it a secret for a few weeks, let alone 17 years.
nittany ram
ModeratorBy the official version do you mean that al Qaeda carried out the attacks under the command of Osama Bin Laden?
Isn’t Bin Laden recorded on video tape talking/bragging about and admitting to the attacks?
nittany ram
ModeratorI’m partial to the music I grew up with. For the most part I stopped listening to new music sometime in the early 90’s.
There are some newer bands I like, but only because they have a retro style reminiscent of the music from my youth.
I like that there are young people who are drawn to older musical styles. Here’s a young blues artist I like doing a Sabbath cover.
nittany ram
ModeratorSo the Rams’ most serious problems, in order:
1. Stopping the run.
2. The uniforms.
3. Edge rush.Yeah, I like the Rams chances to repeat as NFC West champs if they can stay healthy and fix the uniforms.
nittany ram
ModeratorWow. Jared Goff leads the Rams to a score more than 50% of the time he takes the field. This is second only to Tom Brady (and barely so).
Garoppolo’s ranking can be dismissed out of hand. He is a 9’er, and therefore a loser, so his statistics do not warrant serious consideration.
Besides, he only had 50 attempts. Very little statistical power compared to the others on the list with far more attempts.
But note how NFL Research conveniently made 50 attempts the cutoff?
9’er lovin’ hacks, the lot of em’.
June 5, 2018 at 1:22 pm in reply to: "Right to Try" is a bait n' switch that will not help the terminally ill… #87072nittany ram
ModeratorThe ‘cruel joke’ of compassionate use and right to try: Pharma companies don’t have to comply…
The ‘cruel joke’ of compassionate use and right to try: Pharma companies don’t have to comply
From my days in medical school, I vaguely remember learning about lysosomal storage disorders. They occupied at most part of a lecture or two in my second-year pathophysiology course. I memorized a few details about these rare diseases in preparation for my board exam, and then never gave them another thought. These diseases were treated by pediatric specialists and wouldn’t be part of my life as a cardiologist.
That changed a few weeks ago when my 28-month-old daughter, Radha, was diagnosed with a lysosomal storage disorder. Now I know far more about these diseases than I did in medical school. I’ve also learned a frustrating fact that no medical school teaches its students: While the FDA has a compassionate use program to allow people access to experimental drugs, it can’t compel a company to provide those drugs. The newly signed “right-to-try” law doesn’t either.
Radha’s birth went perfectly. She was a healthy baby and met all of her developmental milestones — until it came to walking. My wife, Sonal, a pediatric gastroenterologist, recognized this and we had Radha evaluated by several specialists. None thought anything was physically wrong and indicated that she would learn to walk with the help of some physical therapy sessions.
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They initially helped. Then Radha’s progress slowed. Just after her second birthday, additional testing, including an MRI of her brain and spine followed by a genetic analysis, revealed that our daughter had metachromatic leukodystrophy.
Related: Trump signs right-to-try legislation, making controversial measure law of the land
This lysosomal storage disorder is an autosomal recessive genetic disease that interferes with the body’s production of a single enzyme, arylsulfatase A. Not enough arylsulfatase A causes a buildup of fats called sulfatides inside cells. In cells that make myelin, the substance that insulates and protects nerves, an abundance of sulfatides destroys tissue throughout the brain, spinal cord, and other parts of the nervous system.Children with the most severe form of metachromatic leukodystrophy develop symptoms like trouble walking or poor muscle tone before the age of 30 months. Once symptoms appear, the prognosis is grim. Radha’s health will decline rapidly over the next three to six months. She will soon lose her ability to move, speak, see, and eat, and will be prone to seizures. The disease will then plateau for several years, leaving her in a vegetative state and unable to communicate. Our only hope is that she’ll always understand us when we tell her we love her, but we may never know. Most children with metachromatic leukodystrophy don’t survive beyond their 8th birthday.
Because we live in an era of rapid genomic innovation, gene-editing technologies such as CRISPR, proteomics, and rational drug design, I assumed that a disease caused by a single-enzyme deficiency was treatable. In my search for ways to help my daughter, I came across enzyme replacement therapies being developed for a number of conditions, including metachromatic leukodystrophy.
Shire Pharmaceuticals has developed a therapy for the disease and has even found a way to deliver it across the blood-brain barrier, which is no mean feat. The company has even completed a multicenter Phase 1/2 trial of the drug, called SHP-611 (also known as HGT-1110) in Europe, with what appear to be promising results. There was enough of a signal of therapeutic benefit from this trial to move forward with another one, though it appears to be several months to a year away.
Children with metachromatic leukodystrophy who were involved in the original trial have access to the drug as part of an extension of the trial. Radha developed the disease too late to take part in the first trial, and too soon to join the second one (if and when it happens).
Related: ‘Right-to-try’ law intended to weaken the FDA, measure’s sponsor says in blunt remarks
Even so, that discovery gave me hope. It meant that Radha should qualify for what the Food and Drug Administration calls its expanded access program, also known as compassionate use. It governs the use of an investigational medicine that has not been approved by the FDA outside of a clinical trial.Here’s how it is supposed to work. A physician caring for a patient with a terminal illness who has exhausted all other treatment options and isn’t eligible for a clinical trial appeals to the pharmaceutical company to provide an investigational drug that has undergone at least a Phase 1 trial, which studies the safety of a drug. If the pharmaceutical company agrees, the treating physician applies to the FDA for approval for expanded access to the investigational drug.
Thanks to policy changes at the FDA, it has become easier than ever for physicians seek access to investigational drugs. The application form has been significantly simplified and now only one member of a facility’s institutional review board needs to sign off on the petition. The FDA approves more than 95 percent such requests, and does so swiftly, usually in a matter of a few days.
Radha’s physicians followed Shire’s protocol for applying for compassionate use exactly as directed on the company’s website. Within a day or two, their request was denied, without any legitimate medical reason given.
With my daughter’s life on the line, I shamelessly used every contact and connection I have to reach someone at Shire to ask about compassionate use of SHP-611. When that effort yielded no responses, I called and emailed the current and former FDA commissioners, the head of the pharmaceutical trade association, PhRMA, the former CMO of a major pharmaceutical company, and even the dean of the medical school I attended. Most were cordial, even supportive.
Sonal and I even started a Change.org petition to help us nudge Shire to give Radha and her doctors compassionate access to SHP-611.
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All of our efforts to get answers from Shire have been repeatedly rebuffed with vague, unsatisfying responses, leaving me to wonder why the company is denying my daughter’s only hope. In fact, Shire has refused to correspond with me directly, and has instructed me to direct questions to it via my daughter’s treating physicians.Large pharmaceutical companies are notoriously risk averse when it comes to expanding access to medications that are still in the testing phase. Many refuse to grant access to investigational drugs outside of clinical trials, and efforts to lobby them to release the medication as part of compassionate use are often rebuffed.
One fear they have is that an adverse event, like an injury or death — even if it is not directly due to the medication — will derail a company’s ability to push a drug forward for FDA approval, something they argue would ultimately undermine efforts to develop drugs that can help other families.
In response to this fear, FDA Commissioner Scott Gottlieb unveiled an updated policy on reporting adverse events that occur during compassionate use. It now requires reporting “only if there is evidence to suggest a causal relationship between the drug and the adverse event.”
Pharmaceutical companies also worry that if an experimental medication is given to one patient through compassionate use, it must be given to all patients who request it. In the case of rare diseases like metachromatic leukodystrophy — in the U.S., only about 60 children develop the late infantile form of the disease each year — this could mean that a company would have trouble enrolling enough patients when it eventually opens a clinical trial.
That’s a valid concern when access to the therapy is not time-sensitive. But in disorders such as the late infantile form of metachromatic leukodystrophy, the disease progresses so fast and irreversibly that patients who are denied access to the medication today will soon be so debilitated that they would not derive any benefit from it if and when it became available via a clinical trial, and so would not be able to enroll in the trial anyway.
Related: Right-to-try bill headed for vote puts bigger burden on FDA to protect patients, Gottlieb says
The push for a federal right-to-try process culminated this week with President Trump signing a new law in a ceremony surrounded by patients with life-threatening illnesses and their families. In theory, this law will let patients and physicians bypass the FDA and go directly to pharmaceutical companies for access to investigational therapies that have undergone early testing. But it doesn’t require pharmaceutical companies to accede to these requests.This new law requires drug companies to report clinical outcomes and adverse events, though it reduces their implications by stating that the FDA should not use this information to delay or adversely affect the approval of investigational drugs. As a physician, I believe that removing federal safeguards for experimental drugs is dangerous, and I believe that adverse events should be reported to the FDA as a way to prevent them from happening to other patients. As a parent desperate to help his daughter in any way I can, though, I hope this bill will allay Shire’s fears and encourage it to give SHP-611 to Radha.
I have never been one to malign pharmaceutical companies because I believe they are our best source of new and improved treatments. Yet Radha’s situation has made me cynical of a system in which pharmaceutical companies cater to investors and the physicians who prescribe their products rather than to the consumers of their therapies. I wish I could say that Shire is an outlier, but a quick internet search shows many similar situations where other pharmaceutical companies have denied compassionate use requests for what amount to business decisions.
Shire’s therapy represents the only reasonable hope for Radha and our family. If the company continues to refuse access to SHP-611 outside of a clinical trial, then why not open a new one? Its previous trial ended 15 months ago and yet there is still no sign of the follow-up trial that Shire claims it is working hard to start as soon as possible.
Much of what we do in medicine is based on analyses of benefits and risks. Shire has produced a drug that in early testing demonstrated safety with enough benefit to push forward follow-up trials. In Radha’s case, the potential benefits of SHP-611 clearly outweigh the risks, but only if we get the drug to her soon, before her condition deteriorates further.
Compassionate use and right-to-try are billed as ways to give hope to patients who have exhausted all other options. From Radha’s perspective, they are nothing more than a cruel joke, dangling a potential lifesaving therapy just out of her reach.
Vibhav Rangarajan, M.D., is a fellow in advanced cardiovascular imaging at Northwestern University’s Feinberg School of Medicine.
About the Author
Vibhav Rangarajan
vibhav@gmail.com
@vsranganittany ram
ModeratorIt’s a SKULL. A deceased Ram.
But…I guess that’s what I should expect from a 9ers fan.
It’s not a ‘skull’. It’s a stylized depiction of the head of a Ram reminiscent of the English renaissance.
http://ica.themorgan.org/zoom/default.asp?id=m81.040r
I guess I shouldn’t be surprised that zooey kaepernick has an issue with a Rams logo, especially when it’s the most iconic logo in the history of sports.
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This reply was modified 6 years, 10 months ago by
nittany ram.
nittany ram
ModeratorThats not a real book is it? I will assume its a joke.
w
vIt’s all too real.
Link: https://www.harpercollins.com/9780062749598/the-faith-of-donald-j-trump/
Didn’t you say you were looking for comedy books?
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This reply was modified 6 years, 10 months ago by
nittany ram.
nittany ram
ModeratorNah, I love this logo. Best logo in sports.
nittany ram
ModeratorOther really short books…
“Protecting Your Franchise Quarterback” by Mike Martz
– with a Forward by Mark Bulger where he recounts his experiences throwing from a clean pocket…nittany ram
Moderator“Rams told Nike don’t touch horn design on helmets…”
Well at least the corporate weasels got ‘that’ right.
When the biosphere dies,
and the earth is a silent dystopian desert,
and only iguanas and crows and zombies roam the land,
I hope a limping, broken-legged zombie or two are wearing proper Ram helmets.
Blue and White, preferably.w
vI feel pretty confident in guessing that approximately 17,687 people out of the 3,500 people they surveyed told them to fucking leave the horns the fuck alone.
Well, Nike shouldn’t leave the horns completely alone.
They need to make them thicker.
The thin, wispy horns they currently have are an affront to the memories of Tank Younger and Dan Towler.
Now THAT’S a set of horns you can set your watch to.
June 3, 2018 at 11:14 am in reply to: Trump lawyers sent bombshell memo to Mueller in January #86992nittany ram
ModeratorTrump wants to turn the US into the same sort of autocracy that his hero, Putin, presides over.
And nearly half the country is cheering him on.
Hamilton’s Grand Experiment in democracy is boiling over the edge of the beaker and burning holes in the bench top.
nittany ram
ModeratorSo, msnbc has a story about a boy who raised $6000.00 with his lemonade stand to pay for his terminally ill brother’s medical bills. They reported it as an inspirational story.
However, the story’s not inspirational. It’s a tragedy and should be reported as such. It’s a tragedy because a family has to figure out a way to pay for the medical bills of a dying child. The horrible grief isn’t a big enough price to pay.
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